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FINDINGS: UCLA AIDS Institute researchers successfully removed CCR5 — a cell receptor to which HIV-1 binds for infection but which the human body does not need — from human cells. Individuals who naturally lack the CCR5 receptor have been found to be essentially resistant to HIV.

Using a humanized mouse model, the researchers transplanted a small RNA molecule known as short hairpin RNA (shRNA), which induced RNA interference into human blood stem cells to inhibit the expression of CCR5 in human immune cells.

IMPACT:The findings provide evidence that this strategy can be an effective way to treat HIV-infected individuals, by prompting long-term and stable reduction of CCR5.

FUNDING:The research was funded by a Rheumatology Fellowship Training grant, the UCLA AIDS Institute, the UCLA Center for AIDS Research, the National Institute of Allergy and Infectious Diseases, the National Heart, Lung and Blood Institute, and the National Cancer Institute.

Would this be an equivalent to a CCR5 inhibitor.......like Maraviroc? (one blocks/one removes R5)

I am dual-tropic (X4/R5). I was wondering if this therapy would work for individuals for those who have X4 as well?

Maraviroc never did a thing for me........Until there's a X4 antagonist to couple with an R5 inhibitor......I have to depend on some other form of therapy........hence, I was wondering if this particular gene/stem cell therapy may work for me one day?

Would this be an equivalent to a CCR5 inhibitor.......like Maraviroc? (one blocks/one removes R5)

I am dual-tropic (X4/R5). I was wondering if this therapy would work for individuals for those who have X4 as well?

Maraviroc never did a thing for me........Until there's a X4 antagonist to couple with an R5 inhibitor......I have to depend on some other form of therapy........hence, I was wondering if this particular gene/stem cell therapy may work for me one day?

This basically does what Maraviroc does but at the level of gene snipping so it has the potential to essentially re-jig the immune system, so to speak.

There are other drugs and treatments in development that target X4-tropic virus but one thing that should give you hope is that the patient who was cured of HIV and leukemia (can you imagine having both HIV and leukemia and getting cured of both?@!) in Gemany through a bone marrow transplant with donor stem cells that were missing the CCR5 co-receptor had dual-tropic virus so it's still a mystery why the therapy worked for him . . .but it did. They can't find HIV in his system after two years and his levels of HIV antibodies are decreasing to the point that doctors are predicting he will test HIV-negative at some point.

The patient’s form of the virus, X4, is not typically affected by the CCR5 resistance! This leads to some major questions: why would a CCR5 donor’s stem cells effectively treat a patient with X4 HIV? Is it something special about stem cells? Do we not understand the CCR5 resistance? Do we not understand the X4 form of the virus? Is there a completely unknown factor affecting the results (chemotherapy, HIV medications… solar flares?)

.Hopefully, manipulating the R5 can be done one day without the risk of a Bone Marrow Transplant......Encouraging, given that the patient was dual-tropic........

That's what much of the gene therapy is attempting to do. The success of the treatment with the patient in Germany serves as a proof of principle for gene therapy being able to achieve the same result without the need of a transplant.